Polycystic kidney disease (PKD) is a common inherited human nephropathy that affects approximately 1:1000 of the worldwide population and is the third-most common cause of end stage renal failure. Renal cysts progressively develop in patients afflicted with PKD, and are associated with complications such as cyst infection and hemorrhage, renal stones, and pain. PKD largely results from the Autosomal Dominant Polycystic Kidney Disease (ADPKD), which is attributed to heterozygous mutations in either the Pkd1 or Pkd2 gene. These genes respectively encode the large, membrane-associated proteins Polycystin-1 (PC1) and Polycystin-2 (PC2). In PKD, cyst formation and the associated symptoms may be caused by loss of polycystin function.
No effective treatment for PKD or other cyst-forming disease is currently known. Previous unsuccessful attempts at treatment have included increasing the levels of Tissue Necrosis Factor-alpha (TNF-alpha) in a patient. Accordingly, there exists, inter alia, a need for treating and/or ameliorating the symptoms of PKD and other cyst-forming diseases.